Laronidase
- Generic Name
- Laronidase
- Brand Names
- Aldurazyme
- Drug Type
- Biotech
- CAS Number
- 210589-09-6
- Unique Ingredient Identifier
- WP58SVM6R4
- Background
Human recombinant alpha-L-iduronidase, 628 residues (mature form), produced by recombinant DNAtechnology in a Chinese hamster ovary cell line. Laronidase is a glycoprotein with a molecular weight of approximately 83 kD. The predicted amino acid sequence of the recombinant form, as well as the nucleotide sequence that encodes it, are identical to a polymorphic form of human a-L-iduronidase. It contains 6 N-linked oligosaccharide modification sites.
- Indication
For the treatment of mucopolysaccharidosis
- Associated Conditions
- Mucopolysaccharidosis Type I (MPS I)
BioMarin Acquires Inozyme Pharma for $270 Million, Adding Late-Stage Enzyme Therapy for Rare Genetic Disorder
• BioMarin Pharmaceutical has entered into a definitive agreement to acquire Inozyme Pharma for $270 million, strengthening its enzyme therapy portfolio with the addition of INZ-701.
• INZ-701 is a Phase 3 enzyme replacement therapy being developed for ENPP1 Deficiency, a rare genetic condition affecting blood vessels, soft tissues, and bones with no currently approved treatments.
• The first pivotal data readout for INZ-701 in children is expected in early 2026, with potential regulatory approval and market launch in 2027.
Leap Therapeutics Halves Workforce and Refocuses Cancer Drug Development Amid Market Challenges
• Leap Therapeutics has announced a significant restructuring, reducing its workforce by approximately 50% and narrowing the development focus of its lead cancer drug candidate in response to challenging market conditions.
• The strategic pivot aims to extend the company's cash runway while concentrating resources on the most promising clinical applications of its lead oncology asset, potentially improving its chances for regulatory success.
• This move follows similar restructuring trends across the biotech sector, with companies like Arcturus, NGM Bio, and Erasca all recently announcing staff reductions and pipeline reprioritizations to navigate the difficult funding environment.
Regenxbio Expands MPS I Gene Therapy Trial After Initial Data Review
Regenxbio has decided to expand its phase 1/2 trial for RGX-111, a gene therapy targeting mucopolysaccharidosis type I (MPS I), following promising initial data showing clinical activity. The therapy aims to deliver a functional copy of the IDUA gene to the central nervous system, offering a potential one-shot treatment for this rare inherited disease.
Sanofi's Q3 Sales Surge 15.7% Driven by Vaccines and Dupixent; 2024 EPS Guidance Raised
• Sanofi reported a robust 15.7% sales growth in Q3, fueled by strong vaccine sales and the continued success of Dupixent, with sales up 23.8% to €3,476 million.
• Pharma launches, including ALTUVIIIO, Nexviazyme, and Rezurock, saw a significant 67.1% increase, contributing €727 million to the overall sales growth.
• The company's pipeline advanced with four regulatory approvals, including Dupixent for COPD in the US and China, and four positive Phase 3 data readouts, including tolebrutinib for nrSPMS.
• Based on strong Q3 business performance, Sanofi has raised its 2024 business EPS guidance to at least a low single-digit percentage growth at CER.
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